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Endometriosis Treatment: Time for “Precision Medicine?”

Endometriosis – a condition where uterine lining tissue grows outside of the uterus – affects approximately 10% of reproductive-aged women, 45% of infertility patients, and 80% of women with chronic pelvic pain. The associated pain can be significant and even debilitating. The first-line treatment for women with endometriosis-related pain is hormonal and specifically progestin (progesterone-like) therapy.

A recent study published in the Journal of Clinical Endocrinology & Metabolism from Yale University School of Medicine has shown that the effectiveness of progestin therapy depends on whether a woman's endometriosis-related lesions have the progesterone receptor (PR) present.

The investigators tested the endometriotic lesions of 52 women who had undergone surgical evaluation for endometriosis at Yale New Haven Hospital for their PR status and there was a significant association between PR status and responsiveness to progestin therapy. Those whose endometriotic lesions were PR-positive responded considerably better to the progestin therapy, while those whose lesions were PR-negative found little to no relief from progestin therapy alone.

The research team concluded that knowing the presence or absence of PRs may help in the development of new, individually-targeted, precision-based approaches to treating endometriosis. First author, Dr. Valerie Flores, stated, "Receptor status in endometriosis could be used in a manner analogous to the use of estrogen/progesterone receptor status in breast cancer for tailoring hormonal-based regimens." A targeted approach to endometriosis management may avoid some of the present “trial and error” methods involved with therapy.

This work is the first step in the right direction toward precision medicine in treatment of endometriosis. Further work should focus on other hormonal receptors on endometriotic lesions and ultimately, perhaps the patient’s genome with respect to specific hormonal and other therapies.

Reference: Valerie A Flores, Arne Vanhie, Tran Dang, Hugh S Taylor. Progesterone Receptor Status Predicts Response to Progestin Therapy in Endometriosis. The Journal of Clinical Endocrinology & Metabolism, 2018; DOI: 10.1210/jc.2018-01227

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